Dr.Suren Movsisyan🇪🇺

Some people don't just talk about making a difference. They dedicate their time, energy, and expertise to it. Nina Kilbride is one of those people. I've known Nina for the past 2–3 years, and her determination to make a difference in the PKU and rare-disease community has always stood out to me. She's a lawyer, a founder, and a mother of a child with PKU. What I deeply respect is how she's channelled her personal connection into something bigger: raising awareness, supporting families, and pushing for better research and solutions. Rare diseases deserve more attention, funding, and scientific progress. Research can be challenging when patient populations are small and commercial incentives are limited, but every person living with a rare condition deserves to be heard and supported. I believe AI can help researchers explore new possibilities, analyse complex data, and accelerate parts of the drug discovery process. It won't solve everything overnight, but combining technology with dedicated researchers and people who genuinely care could open new doors for rare-disease communities. That's also why I want to ask you all to check out Phebe: phebe.life/ If you care about PKU, rare diseases, or making life a little easier for affected families, please take a moment to explore it. The more people who understand these challenges and support meaningful initiatives, the better. Nina, I have huge respect for your vision, your persistence, and the work you're doing for the PKU community. Your commitment is a reminder that meaningful change starts with people who care enough to keep showing up. I hope we see more research, better treatment options, and one day, breakthroughs that change the lives of families affected by PKU and other rare diseases. Thank you for continuing to push for that future. ❤️

The Phebe Founding 100: a membership campaign for people with PKU and the people who carry the PAH gene. Phebe is free. Join as a founding member. app.phebe.life/signup #PKU #RareDisease

Acting @FDA Commissioner Kyle Diamantas, @FDA_KyleD, sat down for a fireside chat with #NORD President and CEO Pam Gavin to kick off the 2026 #NORDSummit, where he shared that the #FDA is committed to modernizing its review process and is relying on #RareDisease patient voices to help shape its fit-for-purpose regulatory framework. We appreciated the opportunity to break down what that will entail, and we're excited to be a partner in this crucial, timely mission.

🎉 This week is E.S.PKU week! Sharing moments from our 40th conference in Copenhagen? 🇩🇰 📸 #ESPKU2026 – photos & videos 📝 #ESPKUConference2026 – conference posts 🎉 #40Conferences1Community – celebrating our anniversary and community! Share your experience!

Another (again still small) study showing Phe-Tyrosine ratio is relevance; reason: Tyrosine influences neurotransmitter Dopamine; ppl who have #pku #phenylketonuria don't convert Phe into Tyrosine. One reason why full dose protein substitute is VITAL😎😎😎 onlinelibrary.wiley.com/doi/…

At the @RareDiseases conference with @FDA_KyleD discussing integrating patient voices early. I’m old enough to remember when we were not welcome at the table. Thank you for the leadership!

Our #NORDSummit CEO Leadership Dialogue, moderated by @CNBC's @BeckyQuick, included many important takeaways like this one from @BioMarin CEO Alexander Hardy. Large #biotech companies like his make it a priority to acquire smaller, emerging companies who are doing pioneering work on #RareDiseases. Their larger infrastructure and longer-standing relationships can help secure #FDA approval and scale manufacturing to the next level.

At the #NORDSummit, @CNBC's @BeckyQuick moderated a dialogue with the CEOs of NORD, @BioMarin, @JazzPharma, @OtsukaUS in which she challenged each of them to state their "wish list" for removing barriers to care for rare disease patients. Here were some of their answers: • Cohesive patient data sets powered by foundations and advocacy groups • More funding for organizations like #NORD to sustain that infrastructure • Expanded newborn screening for rare, genetic conditions • Improved medical awareness and education • Diagnostic codes for more rare diseases • More regulatory flexibility and fit-for-purpose standards for highly heterogenous diseases • Consistency and predictability across #FDA review teams • Exemptions from tariffs for orphan drug manufacturing The overarching theme was that, to scale up #RareDisease research and treatment development, we need laws and regulatory policies that address the challenges patient communities and #biotech companies are facing, a #healthcare system with improved diagnostic capabilities, and well-funded patient organizations who can lead research efforts in their communities, in partnership with industry companies.